CNS Pharmaceuticals Secures Orphan Drug Designation for TPI 287 in Brain Cancer Treatment
• CNS Pharmaceuticals has successfully acquired Orphan Drug Designation from Cortice Biosciences for TPI 287, a novel abeotaxane with potential to cross the blood-brain barrier for treating brain tumors.
• In early clinical trials, TPI 287 combined with bevacizumab showed promising efficacy against glioblastoma multiforme (GBM), with 3 complete responses and 9 partial responses among 23 evaluable patients.
• The company plans to begin patient enrollment for a Phase 2 study by the end of 2025, with the Orphan Drug status potentially providing seven years of market exclusivity upon approval.
Coave Therapeutics Unveils coAAV-CSF-01: A Breakthrough Gene Therapy Vector for CNS Diseases
• Coave Therapeutics has launched coAAV-CSF-01, a novel CNS-targeted gene therapy vector showing up to 10,000-fold increased transgene expression in key brain regions compared to AAV9 in non-human primates.
• The new vector demonstrates comparable CNS biodistribution at one-fifth the dose of AAV9, with significantly reduced peripheral transduction and improved safety profile following intra-cerebrospinal fluid administration.
• Developed using Coave's proprietary ALIGATER™ platform, the breakthrough technology addresses key limitations in gene therapy delivery and shows promise for treating neurodegenerative and neurodevelopmental CNS disorders.
FDA Grants Breakthrough Therapy Designation to BrainChild Bio's CAR T-Cell Therapy for Fatal Pediatric Brain Tumors
• The FDA has granted Breakthrough Therapy Designation to BrainChild Bio's B7-H3 targeting CAR T-cell therapy (BCB-276) for diffuse intrinsic pontine glioma (DIPG), based on promising survival data from a Phase 1 trial.
• DIPG affects approximately 300 children annually in the US and is uniformly fatal, with current standard treatment offering only about 11 months median survival from diagnosis.
• BrainChild Bio is preparing to advance BCB-276 in a Phase 2 pivotal registration trial to support a potential Biologics License Application, following alignment with the FDA at a recent Type B meeting.
FDA Approves VALTOCO Nasal Spray for Seizure Clusters in Children Ages 2-5
• The FDA has approved Neurelis' VALTOCO (diazepam nasal spray) for treating seizure clusters in patients as young as 2 years old, expanding its previous indication to include younger children.
• Clinical trials demonstrated VALTOCO's safety and efficacy in children aged 2-5, with 31 of 35 enrolled patients completing the 180-day safety period and no treatment-related serious adverse events reported.
• The nasal spray formulation provides a significant alternative to rectal diazepam, addressing a major unmet need for caregivers managing seizures in young children.
Wave Life Sciences to Seek FDA Approval for Duchenne Muscular Dystrophy Drug Following Promising Phase 2 Results
• Wave Life Sciences will pursue FDA accelerated approval for WVE-N531, an exon 53 skipping therapy for Duchenne muscular dystrophy, following positive Phase 2 trial results showing increased dystrophin production.
• The experimental treatment demonstrated substantial improvements in muscle health with a statistically significant improvement in "time to rise" functional tests compared to historical controls through 48 weeks of treatment.
• If approved, WVE-N531 could offer a potential monthly dosing regimen and become a new therapeutic option for approximately 8-10% of Duchenne patients with specific genetic mutations amenable to exon 53 skipping.
Five-Year CROWN Trial Data Establishes Lorlatinib as First-line Standard for ALK+ NSCLC
• Extended follow-up data from the phase 3 CROWN trial demonstrates superior efficacy of lorlatinib over crizotinib, with median progression-free survival not yet reached at 60.2 months.
• Strategic dosing approach starting at 50mg and gradually increasing to manage neurocognitive side effects is recommended, differing from the standard 100mg daily dosing in prescribing information.
• Approximately 60% of patients may experience neurocognitive adverse events, requiring careful monitoring and proactive management through dose optimization.
NeuroTherapia and Sinaptica Advance Alzheimer's Therapies into Phase 2 Trials
• NeuroTherapia's NTRX-07, an oral neuroinflammatory inhibitor, receives EMA approval for a Phase 2 trial in Alzheimer's disease, aiming to improve neuronal function.
• Sinaptica Therapeutics initiates a Phase 2 trial for its second-generation SinaptiStim® system, combining rTMS and transcranial alternating current stimulation for early Alzheimer's.
• Sinaptica's dual stimulation technology shows potential for enhanced efficacy and reduced treatment time, building on positive Phase 1 data in healthy volunteers.
Dyne Therapeutics' DYNE-101 Shows Promise in DM1 Trial, FDA Grants Fast Track Designation
• Dyne Therapeutics' DYNE-101 demonstrates compelling results in Phase 1/2 ACHIEVE trial for myotonic dystrophy type 1 (DM1).
• The FDA grants Fast Track designation to DYNE-101, expediting its development and regulatory review process.
• Dyne plans to initiate a global Registrational Expansion Cohort, aiming for U.S. Accelerated Approval submission in H1 2026.
• DYNE-251 for Duchenne muscular dystrophy (DMD) is also advancing, with potential regulatory submissions expected in early 2026.
HOPE Therapeutics to Acquire Kadima Neuropsychiatry Institute as Flagship for Global Interventional Psychiatry Network
• NRx Pharmaceuticals subsidiary HOPE Therapeutics announces planned acquisition of Kadima Neuropsychiatry Institute to serve as the flagship for an international network of interventional psychiatry clinics treating depression and PTSD.
• Dr. David Feifel, founder of Kadima and Professor Emeritus at UC San Diego, will join HOPE as Chief Medical Innovation Officer, bringing three decades of expertise in advanced interventional treatments for psychiatric disorders.
• The acquisition aligns with HOPE's strategy to develop multi-modal treatment approaches combining ketamine therapy with transcranial magnetic stimulation and digital therapeutics, supported by $27 million in anticipated funding.
Chemo-Free Ponatinib and Blinatumomab Show Promise in Ph+ ALL
• A chemotherapy-free regimen of ponatinib and blinatumomab demonstrates high rates of deep minimal residual disease (MRD) negativity in newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).
• The regimen resulted in an 88% 3-year overall survival rate, significantly reducing the need for stem cell transplants in this patient population.
• Elevated white blood cell count at diagnosis was identified as a predictor of relapse, prompting investigation into novel consolidation strategies like CAR T-cell therapy for high-risk patients.
• Genomic sequencing identified VPREB1 deletion and CNS disease as potential indicators of relapse risk, warranting further investigation.
BioAge Labs Shifts Focus to NLRP3 Inhibitor After Azelaprag Trial Discontinued
• BioAge Labs has discontinued the Phase 2 STRIDES trial of azelaprag due to liver transaminitis observed in some participants, prioritizing patient safety.
• The company is shifting focus to its NLRP3 inhibitor program, with IND submission anticipated in the second half of 2025, targeting neuroinflammation.
• BioAge continues to leverage its discovery platform, collaborating with Novartis and Lilly to identify novel therapeutic targets related to aging biology.
• Despite the setback with azelaprag, BioAge's financial position remains strong, with cash runway extending beyond 2029, supporting ongoing research and development.
Zorifertinib Approved in China as First-Line EGFR TKI for NSCLC with CNS Metastases
• Alpha Biopharma's zorifertinib receives NMPA approval in China for first-line treatment of NSCLC with EGFR mutations and CNS metastases.
• The EVEREST trial demonstrated zorifertinib's superior ability to control intracranial lesions, reducing the risk of intracranial progression/death by 37%.
• Zorifertinib is the first EGFR-TKI designed as a non-substrate for blood-brain barrier efflux proteins, enhancing its penetration into the brain.
• The approval marks a significant advancement in treating lung cancer patients with brain metastases, addressing a critical unmet need.
Lantern Pharma Expands LP-300 Trial to Taiwan for Never-Smoker NSCLC
• Lantern Pharma has dosed the first patient in Taiwan for its Phase 2 HARMONIC trial, evaluating LP-300 in never-smoker NSCLC patients.
• The trial expansion into Taiwan is significant due to the high proportion of never-smoker lung cancer cases in the region, over 50% of new diagnoses.
• Preliminary results from the trial's safety lead-in cohort showed an 86% clinical benefit rate and a 43% objective response rate.
• The HARMONIC trial is evaluating LP-300 in combination with carboplatin and pemetrexed for patients who have progressed after TKI treatment.
FDA Grants Accelerated Approval to Tarlatamab for Previously Treated Extensive-Stage Small Cell Lung Cancer
• Tarlatamab (Imdelltra) receives accelerated FDA approval for extensive-stage small cell lung cancer (SCLC) post-platinum-based chemotherapy.
• The approval was based on the DeLLphi-301 study, which demonstrated a 40% objective response rate among evaluable patients.
• The median duration of response in the study was 9.7 months, with a significant proportion of responses lasting over 6 months.
• Common adverse events included cytokine-release syndrome, fatigue, and pyrexia, necessitating careful monitoring and management.
FDA Accepts Satsuma and SNBL's NDA Resubmission for STS101 Migraine Treatment
• The FDA has accepted for review Satsuma Pharmaceuticals and SNBL's resubmitted NDA for STS101, a dihydroergotamine nasal powder, for acute migraine treatment.
• The PDUFA date is set for April 30, 2025, offering hope for the nearly 40 million Americans suffering from migraine, especially women in their 20s to 40s.
• STS101 is designed for quick self-administration, leveraging a proprietary nasal delivery device for rapid DHE absorption and sustained plasma concentrations.
• The resubmission addresses FDA's previous concerns related to formulation, with no additional clinical trials requested, marking a significant step toward potential approval.
Trastuzumab Deruxtecan Shows Promise in HER2+ Breast Cancer with Brain Metastases
• Trastuzumab deruxtecan (T-DXd) demonstrates substantial and durable clinical activity in HER2-positive advanced breast cancer patients, including those with brain metastases.
• The DESTINY-Breast12 trial showed a 12-month progression-free survival rate of 61.6% in patients with baseline brain metastases treated with T-DXd.
• Central nervous system overall response rate was 71.7% in patients with measurable CNS disease, indicating significant intracranial activity of T-DXd.
• Safety profile of T-DXd was consistent with previous reports, though interstitial lung disease remains an important risk, especially with concomitant steroid use.
RYBREVANT® Plus Lazertinib Shows Significant PFS Improvement in EGFR-Mutated NSCLC
• The Phase 3 MARIPOSA study demonstrated that RYBREVANT® (amivantamab-vmjw) plus lazertinib significantly improved progression-free survival (PFS) compared to osimertinib in first-line EGFR-mutated NSCLC.
• The combination therapy reduced the risk of disease progression or death by 30% compared to osimertinib, establishing a potential new standard of care.
• An interim overall survival (OS) analysis showed a favorable trend for the RYBREVANT® and lazertinib combination, with ongoing monitoring to determine statistical significance.
• The safety profile of the combination was consistent with individual treatments, with manageable adverse events and infrequent discontinuations, supporting its clinical utility.
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